Hyperbaric oxygen therapy is well tolerated and effective for ulcerative colitis patients hospitalized for moderate-severe flares: a phase 2A pilot multi-center, randomized, double-blind, sham-controlled trial

Am J Gastroenterol. 2018 Oct;113(10):1516-1523. doi: 10.1038/s41395-018-0005-z. Epub 2018 Feb 16.

Abstract

Background: Hyperbaric oxygen therapy (HBOT) markedly increases tissue oxygen delivery. Case series suggest it may have a potential therapeutic benefit in ulcerative colitis (UC). We investigated the therapeutic potential of HBOT as an adjunct to steroids for UC flares requiring hospitalization.

Methods: The study was terminated early due to poor recruitment with 18 of the planned 70 patients enrolled. UC patients hospitalized for moderate-severe flares (Mayo score ≥6, endoscopic sub-score ≥2) were block randomized to steroids + daily HBOT (n = 10) or steroids + daily sham hyperbaric air (n = 8). Patients were blinded to study assignment, and assessments were performed by a blinded gastroenterologist. Primary outcome was the clinical remission rate at study day 5 (partial Mayo score ≤2 with no sub-score >1). Key secondary outcomes were: clinical response (reduction in partial Mayo score ≥2, rectal bleeding sub-score of 0-1) and progression to second-line therapy (colectomy or biologic therapy) during the hospitalization.

Results: A significantly higher proportion of HBOT-treated patients achieved clinical remission at study day 5 and 10 (50 vs. 0%, p = 0.04). HBOT-treated patients less often required progression to second-line therapy during the hospitalization (10 vs. 63%, p = 0.04). The proportion requiring in-hospital colectomy specifically as second-line therapy for medically refractory UC was lower in the HBOT group compared to sham (0 vs. 38%, p = 0.07). There were no serious adverse events.

Conclusion: In this small, proof-of-concept, phase 2A trial, the use of HBOT as an adjunctive therapy to steroids for UC patients hospitalized for moderate-severe flares resulted in higher rates of clinical remission, and a reduction in rates of progression to second-line therapy during the hospitalization. Larger well-powered trials are needed, however, to provided definitive evidence of therapeutic benefit.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study
  • Randomized Controlled Trial

MeSH terms

  • Adult
  • Biological Products / administration & dosage*
  • Colectomy / statistics & numerical data*
  • Colitis, Ulcerative / diagnosis
  • Colitis, Ulcerative / therapy*
  • Combined Modality Therapy / adverse effects
  • Combined Modality Therapy / methods
  • Disease Progression
  • Double-Blind Method
  • Drug Resistance
  • Female
  • Gastrointestinal Hemorrhage
  • Glucocorticoids / administration & dosage*
  • Glucocorticoids / adverse effects
  • Hospitalization
  • Humans
  • Hyperbaric Oxygenation / adverse effects*
  • Male
  • Middle Aged
  • Pilot Projects
  • Proof of Concept Study
  • Remission Induction / methods
  • Severity of Illness Index
  • Symptom Flare Up
  • Treatment Outcome
  • Young Adult

Substances

  • Biological Products
  • Glucocorticoids